Gene Editing Pioneer Sangamo competitors & alternatives

Similar biotech company

CRISPR Therapeutics AG (CRSP) is a biotechnology firm dedicated to pioneering gene-based medicines for severe diseases. The company achieves this through its exclusive Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) technology, which enables precise and targeted alterations to an organism's genetic code. Its extensive pipeline includes therapeutic candidates spanning multiple medical areas, such as blood disorders (hemoglobinopathies), various cancers (oncology), regenerative medicine, and rare conditions. The company's flagship investigational therapy is CTX001, an ex vivo CRISPR gene-edited treatment. This therapy aims to benefit patients with transfusion-dependent beta-thalassemia or severe sickle cell disease by modifying their own hematopoietic stem cells to markedly boost the production of fetal hemoglobin within red blood cells. CRISPR Therapeutics is also advancing several other genetically engineered allogeneic (donor-sourced) CAR-T investigational therapies: CTX110, designed to combat cluster of differentiation 19-positive malignancies; CTX120, which targets B-cell maturation antigen for multiple myeloma that has relapsed or proven resistant to previous treatments; and CTX130, focused on Cluster of Differentiation 70 for a spectrum of solid tumors and blood cancers. Furthermore, the company is developing VCTX210, an immune-evasive, gene-edited stem cell-derived product candidate for treating type 1 diabetes. It is also pursuing various in vivo gene-editing initiatives aimed at addressing disorders affecting the liver, lungs, muscles, and central nervous system. The company has forged strategic alliances with significant partners, including Bayer Healthcare LLC, Vertex Pharmaceuticals Incorporated, ViaCyte, Inc., Nkarta, Inc., and Capsida Biotherapeutics. CRISPR Therapeutics AG was established in 2013 and maintains its headquarters in Zug, Switzerland.

Biotechnology

Excision BioTherapeutics Inc. is a San Francisco-based clinical-stage biotechnology company focused on developing advanced gene editing therapeutics to treat infectious diseases.

Biotechnology

Beam Therapeutics Inc., founded in 2017 and based in Cambridge, Massachusetts, operates as a pioneering biopharmaceutical firm. Its core mission involves engineering precise genetic remedies to tackle a spectrum of severe human ailments, primarily within the United States. The company's developmental portfolio features several key candidates: BEAM-101 is being advanced to treat both sickle cell disease and beta thalassemia. BEAM-102 is specifically designed for addressing sickle cell disease. BEAM-201, an allogeneic chimeric antigen receptor T-cell therapy, is under investigation for individuals suffering from relapsed or refractory T-cell acute lymphoblastic leukemia. BEAM-301 is a liver-targeted candidate aimed at patients afflicted with Glycogen Storage Disease Type Ia. Beyond these flagship programs, Beam Therapeutics is also engaged in devising treatments for alpha-1 antitrypsin deficiency, various ocular conditions, and other disorders impacting the liver, muscular system, and central nervous system. To further its research and development objectives, Beam Therapeutics has forged numerous strategic alliances and partnerships. These include collaborations with Boston Children's Hospital; a research and clinical trial agreement with Magenta Therapeutics, Inc.; a licensing deal with Sana Biotechnology, Inc.; and a research partnership with the Institute of Molecular and Clinical Ophthalmology Basel. Additionally, the company maintains research collaborations with both Pfizer Inc. and Apellis Pharmaceuticals, Inc., alongside a comprehensive collaboration and license agreement with Verve Therapeutics, Inc.

Scribe Therapeutics, Inc.SCTX · BiotechAlameda, United States

66% matchPublic

Similar biotech company

Scribe Therapeutics is a clinical-stage biotechnology company that develops genetic therapies for conditions such as heart disease and has pursued public offerings in the United States.

Biotechnology

EdiGene is a clinical-stage biotechnology company founded in 2015 that focuses on translating gene-editing technologies into therapeutic products. Headquartered in Beijing, China, the company also operates research and office facilities in locations such as Shanghai, Guangzhou, and the United States.

Verve Therapeutics, Inc.BiotechnologyUnited States

65% matchPrivate

Biotechnology

Verve Therapeutics, Inc. is a biotechnology company headquartered in Boston, Massachusetts, dedicated to pioneering single-course gene editing medicines for cardiovascular disease.

Splice BioBiotechUnited States

65% match

Similar biotech company

SpliceBio is a clinical-stage genetic medicines company that develops gene therapies using a proprietary Protein Splicing platform. The company focuses on treating diseases caused by mutations in large genes, with a lead clinical program targeting Stargardt disease.

Tessera TherapeuticsBiotechUnited States

65% match

Similar biotech company

Tessera Therapeutics is a biotechnology company developing programmable genetic medicines using its Gene Writing technology, which is based on mobile genetic elements to insert, replace, or modify DNA in cells. It aims to develop treatments for genetic diseases and other serious conditions, serving patients who need durable genetic therapies.

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Editas Medicine, Inc., a clinical stage genome editing company, focuses on developing transformative genomic medicines to treat a range of serious diseases. The company develops a proprietary gene editing platform based on CRISPR technology. Its lead program is EDIT-401, a one-time therapy designed to reduce LDL cholesterol through the upregulation of the LDL receptor to treat hyperlipidemia. The company also develops therapies to treat Sickle cell disease and transfusion-dependent beta thalassemia; and in vivo gene editing medicines indicated for other cells and tissues. It has a research collaboration with Juno Therapeutics, Inc. to develop alpha-beta T-cell experimental medicines for the treatment of solid and liquid tumors, and autoimmune disease. The company was formerly known as Gengine, Inc. and changed its name to Editas Medicine, Inc. in November 2013. Editas Medicine, Inc. was incorporated in 2013 and is based in Cambridge, Massachusetts.

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Intellia Therapeutics, Inc. is a biotechnology firm dedicated to advancing therapeutic treatments through its expertise in genome editing. The company's pipeline includes several in vivo (administered within the body) programs. NTLA-2001 is currently undergoing a Phase 1 clinical trial for transthyretin amyloidosis, while NTLA-2002 targets hereditary angioedema. Additionally, Intellia is developing various other liver-focused therapies for conditions such as hemophilia A and B, hyperoxaluria Type 1, and alpha-1 antitrypsin deficiency. Its ex vivo (processed outside the body) pipeline features NTLA-5001, a candidate for acute myeloid leukemia. The company is also progressing proprietary programs focused on creating engineered cell therapies to address diverse oncological and autoimmune disorders. At the core of Intellia's technology is the Clustered, Regularly Interspaced Short Palindromic Repeats/CRISPR associated 9 (CRISPR/Cas9) system. Intellia Therapeutics, Inc. has established multiple licensing and collaboration agreements. These include a partnership with Novartis Institutes for BioMedical Research, Inc. to engineer hematopoietic stem cells for treating sickle cell disease, and a co-development agreement with Regeneron Pharmaceuticals, Inc. for potential products against hemophilia A and B. The company also collaborates with Ospedale San Raffaele and holds a strategic alliance with SparingVision SAS to develop novel genomic medicines for ocular diseases, leveraging CRISPR/Cas9 technology. The company, initially known as AZRN, Inc., was founded in 2014 and operates from its headquarters in Cambridge, Massachusetts.

Biotechnology

Base Therapeutics is a biotechnology company that develops next-generation gene editing technologies, including its proprietary AccuBase base editing system and enhanced prime editing technology. The company focuses on creating therapeutic solutions for cancer and genetic diseases, alongside engineered immune cell therapies such as BASE-NK and CAR-T products. It serves patients and partners with pharmaceutical and agricultural enterprises by licensing its precision gene editing tools.

Epicrispr BiotechnologiesBiotechnologyUnited States

64% match

Biotechnology

Epicrispr Biotechnologies is a pioneering therapeutics company dedicated to developing advanced genetic medicines. The organization focuses on scientific research, platforms, and clinical pipelines to treat serious diseases.